PB1947: ELTROMBOPAG COMBINED WITH IST AS A MULTICENTER STUDY OF THE FIRST LINE TREATMENT OF CHILDREN WITH SAA.
Authors: L. Yang, H. He, Y. Fang, Y. Yang, S. Lin, G. He, N. Wang
Journal: HemaSphere (2022)

Background: Eltrombopag and Improved Hematopoiesis in Refractory Aplastic was published in the New England Journal of Medicine in 2012 The research found that Eltrombopag monotherapy showed significant hematologic reaction in adult patients with refractory SAA, of which 44%(11/25) patients had clinically significant hematologic performance. Important progress has also been made in the treatment of SAA with Eltrombopag combined with IST: A nonrandomized, historically-controlled study funded by the NHLBI intramural Research Program of the National Institutes of Health compared the efficacy and safety of conventional IST therapy with Eltrombopag in combination with IST for SAA. The research results were published in Eltrombopag Added to Standard Immunosuppression for Aplastic Anemia in 2017 in the New England Journal of Medicine. In this study, a total of 92 SAA patients aged 2 years and above were enrolled, and the results showed that the overall effective rate and complete response rate of Eltrombopag combined with IST in the treatment of SAA were higher than that of traditional IST standard treatment. However, there is no research on Eltrombopag therapy for children SAA in China. The aim of this study was to improve the hematologic response rate and quality, and to prevent late complications such as recurrence and clonal progression, by adding Eltrombopag to standard IST therapy. Aims: To evaluate the efficacy and safety of attriboma combined with IST as first-line treatment for SAA patients, and to study the clonal transformation and the incidence of PNH cloning in attriboma combined with IST patients. Methods: This study was a non-randomized, multi-center, historical controlled study, with traditional IST standard therapy as the historical control. All patients in the experimental group received “IST+ Attribopal” combined therapy, in which ATG dose was 3.5mg. Kg-1.d-1 for a total of 5 days. The initial dose was 1.25 mg · kg-1 · d-1 for 2-5 years old children, 37.5mg · d-1 for 6-11 years old, 75mg · d-1 for 12-18 years old, and continued for 6 months. A total of 31 children were included in the Altrupalpa group and 136 in the historical control group. Results: There were no differences in gender, severity of disease, co-infection before ATG, co-infection within 1 month after ATG, incidence of serum disease, CD3, CD4, CD8, TREG cell count, hemoglobin level, platelet count, neutrophil count and ferritin level between the attripopa group and the historical control group. In terms of the effective rate, the effective rate of attriboma group was 62.5% vs 68.8% (P= 0.548) in June and 86.4% vs 76%(P=0.285) in September, showing no statistical significance. Among them, neutrophil count recovery was better in the attriboma group than in the historical control group at 1 month, 3 months, 6 months, 9 months and 12 months after the application of ATG, the difference was statistically significant (P < 0.05), while platelet and hemoglobin recovery was not different. 2-year EFS 80.4% VS 83.4% (P=0.799) and 2-year OS 93.3% VS 91.3% (P=0.548) in the attriboma group and the historical control group. None of the patients with clone transformation or PNH clone were found in all the follow-up of attriboma group. Summary/Conclusion: There was no difference in infection rate, serum disease rate and overall effective rate between the two groups within 1 month, but the neutrophil count level of the attriboma groups was better than that of the historical control group.